To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.

This is a prospective exploratory phase 2 study designed to evaluate the safety and efficacy of mitapivat in RBC membranopathies and CDAII, a rare sub type of anemia. Nine patients from Princess Margaret who are diagnosed with CDAII will be enrolled to the study. Patients will be in the trial for 57 weeks treatment weeks and a safety follow up week after 30 days from last dose. First 8 weeks will be dose escalating period followed by 48 weeks of fixed dose period. 57th week will be dose tapering week. Data collected from Princes Margaret will be incorporated to the main study conducted in EU for analysis. Overall, approximately 25 patients are expected to be enrolled: Approximately 16 patients at sites in the EU and approximately 9 patients in Canada.

NCT Number	NCT07055243
Lead Sponsor	University Health Network, Toronto Collaborators: Agios Pharmaceuticals, Inc.
Conditions	Anemia
Enrollment	9 participants
Start Date	2025-06-26
Primary Completion	2026-06-30 (estimated)
Study Completion	2027-06-30 (estimated)
Updated on ClinicalTrials.gov	2025-07-08